ATBECHDEDKESFRGBGRITLILUNLSEMCPTIESILTLVFIROMKCYALTRBGCZEEHUPLSK..HRIS..MTNORS..SM..................JBDM Ver 2.0.24 - 1620000/04289951EUROPEAN PATENT APPLICATIONA320240313EP23185302.920201204enenen201962943779 P20191204US201962943797 P20191204US202062972194 P20200210US202063022248 P20200508US202063087582 P20201005US2024031320241120231213202350A61K 48/00 20060101AFI20240206BHEP A61K 31/7088 20060101ALI20240206BHEP C12N 15/115 20100101ALI20240206BHEP C12N 15/64 20060101ALI20240206BHEP A61K 31/7105 20060101ALI20240206BHEP C07K 14/725 20060101ALI20240206BHEP C12N 15/85 20060101ALI20240206BHEP C12N 15/11 20060101ALI20240206BHEP C12N2310/20 20170501 LA20210702BHEP A61K 48/005 20130101 FI20210702BHEP C07K 14/7051 20130101 LI20210702BHEP A61K 48/0008 20130101 LI20210702BHEP A61K 48/0041 20130101 LI20210702BHEP C12N 15/88 20130101 LI20210702BHEP A61K 39/39 20130101 LI20210709BHEP A61K2039/5156 20130101 LA20210709BHEP A61K 39/001112 20180801 LI20210709BHEP A61K 39/0011 20130101 LI20210709BHEP A61K 31/713 20130101 LI20210728BHEP C12N 15/1137 20130101 LI20210827BHEP C12N2310/14 20130101 LA20210827BHEP C12Y 304/21021 20130101 LI20210827BHEP C12N2310/532 20130101 LA20210827BHEP A61K 9/0019 20130101 LI20210914BHEP A61K 9/1272 20130101 LI20210914BHEP A61K 9/1271 20130101 LI20210914BHEP C07K2319/50 20130101 LA20211215BHEP Y02A 50/30 20180101 LA20231211BGEP deZIRKULÄRE RNS-ZUSAMMENSETZUNGEN UND VERFAHRENenCIRCULAR RNA COMPOSITIONS AND METHODSfrMÉTHODES ET COMPOSITIONS D'ARN CIRCULAIRE45B20834039.8392097620201204Orna Therapeutics, Inc.101952611P41243EPD1620 Memorial Drive, 2nd FloorCambridge, MA 02139USGoodman, BrianCambridge, MAUSWesselhoeft, Robert AlexanderCambridge, MAUSHorhota, Allen T.Cambridge, MAUSYang, JunghoonCambridge, MAUSWright, Simon Market al101938771Schlich Ltd 9 St. Catherine's RoadLittlehampton, West Sussex BN17 5HSGBALATBEBGCHCYCZDEDKEEESFIFRGBGRHRHUIEISITLILTLULVMCMKMTNLNOPLPTRORSSESISKSMTR20240313202411

Disclosed herein are circular RNAs and transfer vehicles, along with related compositions and methods of treatment. The circular RNAs can comprise group I intron fragments, spacers, an IRES, duplex forming regions, and/or an expression sequence, thereby having the features of improved expression, functional stability, low immunogenicity, ease of manufacturing, and/or extended half-life compared to linear RNA. Pharmaceutical compositions comprising such circular RNAs and transfer vehicles are particularly suitable for efficient protein expression in immune cells in vivo. Also disclosed are precursor RNAs and materials useful in producing the precursor or circular RNAs, which have improved circularization efficiency and/or are compatible with effective circular RNA purification methods.

P41243EPD1EP23185302.9Orna Therapeutics, Inc.MN20240212C12NA61KC40BC07KA01K
WESSELHOEFT R ALEXANDER ET ALRNA Circularization Diminishes Immunogenicity and Can Extend Translation DurationIn VivoMOLECULAR CELL2019050274310.1016/J.MOLCEL.2019.02.0151097-2765508520XP085676575
Y1-15* the whole document ** in particular the figures *
JESSICA B. FOSTER ET ALPurification of mRNA Encoding Chimeric Antigen Receptor Is Critical for Generation of a Robust T-Cell ResponseHUMAN GENE THERAPYGB2019020130210.1089/hum.2018.1451043-0342168178XP055660199
Y1-15* the whole document *
M. PUTTARAJU ET ALGroup I permuted intron-exon (PIE) sequences self-splice to produce circular exonsNUCLEIC ACIDS RESEARCH19920101202010.1093/nar/20.20.53570305-104853575364XP055622176
A,D1-15* abstract ** page 5358, right-hand column, last paragraph - page 5359, right-hand column, paragraph 1 ** page 5361, left-hand column, paragraph 2 - paragraph 3 ** page 5363, left-hand column, paragraph 1 - right-hand column, paragraph 1 *
LESCA M. HOLDT ET ALCircular RNAs as Therapeutic Agents and TargetsFRONTIERS IN PHYSIOLOGY20181009910.3389/fphys.2018.01262XP055645982
A1-15* page 10, left-hand column, paragraph 3 - right-hand column, paragraph 1 *
R. ALEXANDER WESSELHOEFT ET ALEngineering circular RNA for potent and stable translation in eukaryotic cellsNATURE COMMUNICATIONS201807069110.1038/s41467-018-05096-6XP055622096
A,D1-15* page 2, left-hand column, paragraph 3 - page 5, left-hand column, paragraph 1 ** figures 2,3,6 ** page 6, left-hand column, line 20 - right-hand column, line 6 ** page 8, right-hand column, paragraph 2 - page 9, left-hand column, paragraph 1 ** page 9, left-hand column, paragraph 4 *WO2014186334A1KRUSE ROBERT20141120A1-15* paragraph [0011] - paragraph [0012] ** paragraph [0020] - paragraph [0037] ** paragraph [0098] - paragraph [0099] ** paragraph [0113] - paragraph [0135] ** paragraphs [0106], [0110], [0111] ** figures 1-10 *
CHEN Y GRACE ET ALSensing Self and Foreign Circular RNAs by Intron IdentityMOLECULAR CELL2017061567210.1016/J.MOLCEL.2017.05.0221097-2765page 228XP085140194
A,D1-15* the whole document ** page 228, right-hand column, paragraph 2 ** figures 1,2 *
JAMES C. KACZMAREK ET ALAdvances in the delivery of RNA therapeutics: from concept to clinical realityGENOME MEDICINE201706279110.1186/s13073-017-0450-0XP055464283
A,D1-15* figure 1; tables 1,2 ** page 10, left-hand column, paragraph 1 *
SONJA PETKOVIC ET ALRNA circularization strategies in vivo and in vitroNUCLEIC ACIDS RESEARCH2015020643410.1093/nar/gkv0450305-104824542465XP055488942
A,D1-15* page 2455, right-hand column, paragraph 3 *
B. A. SULLENGER ET ALFrom the RNA world to the clinicSCIENCE20160617352629210.1126/science.aad87090036-807514171420XP055482629
A1-15* abstract ** page 1418, left-hand column, paragraph 2 *Seranski, PeterMunich20240122WO2014186334A120141120AU2014265608A120160107CA2912665A120141120EP2996697A120160323ES2746803T320200306JP6625521B220200108JP2016521133A20160721US2016083747A120160324US2018080041A120180322WO2014186334A120141120